orphan drugs understanding the rare disease market and its dynamics woodhead publishing series in biomedicine

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Orphan Drugs

Author : Elizabeth Hernberg-Ståhl
ISBN : 9781908818393
Genre : Medical
File Size : 69. 58 MB
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This authoritative and comprehensive book makes the reader familiar with the processes of bringing orphan drugs to the global market. There are between 5,000 and 7,000 rare diseases and the number of patients suffering from them is estimated to be more than 50 million in the US and Europe. Before the orphan drug legislation enacted in the US in 1983, there was a limited interest from industry to develop treatment for very small patient groups. One of the difficulties is, of course, that similar levels of investment are needed from a pharmaceutical company to bring a drug to the market for both small and large patient groups. The journey from application of an orphan drug designation to a reimbursed market- approved drug is long and many obstacles occur during the journey. After reading the book, readers will: Understand who the players/stakeholders are in the rare orphan disease field and their specific needs and concerns: patients and patient organizations, researchers and treating physicians within the field, industry, regulatory and reimbursement bodies* Understand the strong partnership between the different players and the various initiatives to improve and increase access to treatment for patients; minimizing the gap between numbers of known diseases, orphan designations, approved drugs and paid drugs. The book also provides short practical case stories from patients and researchers, as well as representatives from industry and authorities on the challenges they came across in developing orphan drugs or getting access to orphan drugs. A comprehensive overview of strategy, key activities and considerations of how to bring an orphan drug from concept to the market and make it available to patients A source of updated information, news and trends for those who are already active in this fast-evolving field Covers the global definitions and the criteria for getting an orphan drug designation in, for example, the US and Europe

A Practical Guide To Rational Drug Design

Author : Sun Hongmao
ISBN : 9780081001059
Genre : Medical
File Size : 71. 12 MB
Format : PDF, Kindle
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This book is not going to be an exhaustive survey covering all aspects of rational drug design. Instead, it is going to provide critical know-how through real-world examples. Relevant case studies will be presented and analyzed to illustrate the following: how to optimize a lead compound whether one has high or low levels of structural information; how to derive hits from competitors’ active compounds or from natural ligands of the targets; how to springboard from competitors’ SAR knowledge in lead optimization; how to design a ligand to interfere with protein-protein interactions by correctly examining the PPI interface; how to circumvent IP blockage using data mining; how to construct and fully utilize a knowledge-based molecular descriptor system; how to build a reliable QSAR model by focusing on data quality and proper selection of molecular descriptors and statistical approaches. A Practical Guide to Rational Drug Design focuses on computational drug design, with only basic coverage of biology and chemistry issues, such as assay design, target validation and synthetic routes. Discusses various tactics applicable to daily drug design Readers can download the materials used in the book, including structures, scripts, raw data, protocols, and codes, making this book suitable resource for short courses or workshops Offers a unique viewpoint on drug discovery research due to the author’s cross-discipline education background Explores the author’s rich experiences in both pharmaceutical and academic settings

Therapeutic Risk Management Of Medicines

Author : Stephen J. Mayall
ISBN : 9781908818270
Genre : Medical
File Size : 57. 49 MB
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Therapeutic risk management of medicines is an authoritative and practical guide on developing, implementing and evaluating risk management plans for medicines globally. It explains how to assess risks and benefit-risk balance, design and roll out risk minimisation and pharmacovigilance activities, and interact effectively with key stakeholders. A more systematic approach for managing the risks of medicines arose following a number of high-profile drug safety incidents and a need for better access to effective but potentially risky treatments. Regulatory requirements have evolved rapidly over the past decade. Risk management plans (RMPs) are mandatory for new medicinal products in the EU and a Risk Evaluation and Mitigation Strategy (REMS) is needed for certain drugs in the US. This book is an easy-to-read resource that complements current regulatory guidance, by exploring key areas and practical implications in greater detail. It is structured into chapters encompassing a background to therapeutic risk management, strategies for developing RMPs, implementation of RMPs, and the continuing evolution of the risk management field.The topic is of critical importance not only to the pharmaceutical and biotechnology industries, but also regulators and healthcare policymakers.Some chapters feature contributions from selected industry experts. An up-to-date practical guide on conceiving, designing, and implementing global therapeutic risk management plans for medicines A number of useful frameworks are presented which add impact to RMPs (Risk Management Plans), together with regional specific information (European Union, United States, and Japan) A comprehensive guide for performing risk management more effectively throughout a product’s life-cycle

Sterility Sterilisation And Sterility Assurance For Pharmaceuticals

Author : Tim Sandle
ISBN : 9781908818638
Genre : Medical
File Size : 27. 49 MB
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Failure to adequately control any microbial challenge associated within process or product by robust sterilisation will result in a contaminated marketed product, with potential harm to the patient. Sterilisation is therefore of great importance to healthcare and the manufacturers of medical devices and pharmaceuticals. Sterility, sterilisation and sterility assurance for pharmaceuticals examines different means of rendering a product sterile by providing an overview of sterilisation methods including heat, radiation and filtration. The book outlines and discusses sterilisation technology and the biopharmaceutical manufacturing process, including aseptic filling, as well as aspects of the design of containers and packaging, as well as addressing the cleanroom environments in which products are prepared. Consisting of 18 chapters, the book comprehensively covers sterility, sterilisation and microorganisms; pyrogenicity and bacterial endotoxins; regulatory requirements and good manufacturing practices; and gamma radiation. Later chapters discuss e-beam; dry heat sterilisation; steam sterilisation; sterilisation by gas; vapour sterilisation; and sterile filtration, before final chapters analyse depyrogenation; cleanrooms; aseptic processing; media simulation; biological indicators; sterility testing; auditing; and new sterilisation techniques. Covers the main sterilisation methods of physical removal, physical alteration and inactivation Includes discussion of medical devices, aseptically filled products and terminally sterilised products Describes bacterial, pyrogenic, and endotoxin risks to devices and products

A Biotech Manager S Handbook

Author : M O'Neill
ISBN : 9781908818157
Genre : Science
File Size : 52. 62 MB
Format : PDF, ePub
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A biotech manager's handbook lays out - in a simple, straightforward manner - for the manager or would-be entrepreneur the basic principles of running a biotech company. Most managers in biotechnology companies are working in their first company or in their first managerial role. Their expertise and experience in the scientific part of the work can be taken as a given but there is a whole range of other skills to be learned and areas of expertise to come to terms with. Small companies do not have big budgets to hire people or time to become an expert in so many areas. The book starts by outlining the state of the biopharmaceutical industry and goes on to explain the importance of planning (no matter what the size of the company). Succeeding chapters deal with the basics of intellectual property, perspectives from a university technology transfer office and how to raise some initial funding from an investor and entrepreneur. No other 'how to' manual exists for this sector Written by a range of expert professionals in each area, all in one book Is the only 'bench to bedside' book covering the whole spectrum of development

Self Management For Actors

Author : Bonnie Gillespie
ISBN : 0972301968
Genre : Performing Arts
File Size : 39. 64 MB
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This book will help you take control of the business side of your creative career. By learning how to build your brand, target your buyers, create content that showcases you best, and pitch like the rockstar you are, you'll build a fulfilling career (wherever you live) that helps rocket you to the next tier.

Pharmaceutical Policy In China

Author : Who Regional Office for Europe
ISBN : 928905039X
Genre : Medical
File Size : 51. 46 MB
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China has a complex pharmaceutical system that is currently undergoing significant reforms. This book provides a comprehensive overview of China's pharmaceutical system and covers key topics such as drug approvals and quality regulation, expenditure trends, pricing and reimbursement, irrational prescribing, traditional Chinese medicine, industrial policy, and the role of hospitals, primary care, and pharmacies.

Early Childhood Counts

Author :
ISBN : 0821345672
Genre : Education
File Size : 65. 75 MB
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Details the preparation, planning, implementation, financing, monitoring, and evaluation of diverse early childhood care programming strategies.

Nutrigenomics And Proteomics In Health And Disease

Author : Yoshinori Mine
ISBN : 0813807174
Genre : Technology & Engineering
File Size : 42. 91 MB
Format : PDF, Mobi
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Part of the Functional Food Science and Technology book series (Series Editor: Fereidoon Shahidi), this book compiles the current science based upon nutrigenomics and proteomics in food and health. Coverage includes many important nutraceuticals (food factors) and their impact on gene interaction and health. Authored by a stellar international team of multidisciplinary researchers, this book acquaints food and nutrition professionals with these new fields of nutrition research and conveys the state of the science to date.

Moral Pressure For Responsible Globalization

Author : Sherrie M. Steiner
ISBN : 9789004365018
Genre : Business & Economics
File Size : 48. 92 MB
Format : PDF
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In Moral Pressure for Responsible Globalization, Steiner offers an account of religious diplomacy with the G8/G7 and G20 to evoke new possibilities to steer globalization in more equitable and sustainable directions in the Age of the Anthropocene.

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